RNA Writing Trans-Splicing for Enhancing Frataxin Translation
Characterization of Fatigue in Patients with Friedreich’s Ataxia by Assessment of Mitochondrial Function in Skeletal and Cardiac Muscle
Correcting Cell-Autonomous Neuroinflammatory Phenotypes in Friedreich’s Ataxia Microglia Using iPSC-Based Approaches
Development and Validation of a New Friedreich’s Ataxia Isogenic Control Mouse
Exploiting Time-Specific Frataxin Inactivation to Unravel Its Role in Peripheral Neurodegeneration in Friedreich Ataxia
Structural Dynamics of the Iron-Sulfur Cluster Assembly Machinery in Patient-Derived FRDA Cells Using Super-Resolution Microscopy
Rescuing frataxin expression by alternative splicing reprogramming with base editing
Deciphering the neurodevelopmental component in Friedreich Ataxia
Investigating epigenetic silencing in Friedreich’s Ataxia
Therapeutic activity of a haematopoietic stem cell delivered tissue penetrating peptide in a Friedreich’s ataxia mouse model